Novartis plans filing for lead CAR-T therapy early next year

Shows complete remission for 82 percent of paediatric and young adult B-cell ALL patients

Novartis has shrugged off the anxieties surrounding CAR-T therapies for cancer after its lead candidate CTL109 showed strong efficacy in a registration trial, setting up marketing applications in early 2017.

All told, 82% of relapsed/refractory paediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL) who were treated with CTL109 went into complete remission in the phase II ELIANA study, and 60% of these were still relapse-free six months later.

The therapy was not without hazard, however, as 48% of patients experienced a recognised side effect of immune-modifying therapies known as cytokine release syndrome (CRS). Novartis said the fever-like condition was managed by clinicians and, while 15% of patients experienced grade 3 neurological and psychiatric events including encephalopathy and delirium, there were no deaths in the trial.

The drugmaker's confidence in the data - reported at the American Society of Hematology (ASH) conference in San Diego - may go some way to calm jitters about the potential of CAR-T, an emerging technology that relies on the use of modified immune cells that are engineered to attack and destroy malignant cells.

Patient deaths in a trial of another candidate in development at Juno Therapeutics - eventually attributed to a cytotoxic drug used to prime the patient for the therapy - raised questions about the safety of the procedure earlier this year. The trial in B-cell ALL resumed with a protocol change, but was halted again two weeks ago after two patients suffered cerebral oedema, one of whom died.

Meanwhile, another CAR-T developer - Kite Pharma - has reported encouraging data with a non-Hodgkin's lymphoma (NHL) candidate and has also said it plans to file for approval in 2017.

Novartis said ELIANA is the first global CAR-T cell therapy trial and would be used to support a regulatory filing in the US early next year, with a marketing application in Europe following later in 2017. CTL109 was awarded PRIME (priority medicine) status by the EMA, one of the first therapies to enter the new expedited approval pathway.

"This first-of-its-kind trial represents exciting progress toward our goal of helping children and young adults with relapsed or refractory B-cell ALL, a patient population with an urgent need for new treatment options," said Bruno Strigini, head of Novartis Oncology.

"We are committed to advancing CTL019 and look forward to working closely with the FDA and EMA in the coming months."